Sumboonnanonda Achra, Sanpakit Kleebsabai, Piyaphanee Nuntawan
Department of Pediatrics, Faculty of Medicine, Siriraj Hospital, Mahidol University, Prannok Road, Bangkoknoi, Bangkok, 10700, Thailand.
Pediatr Nephrol. 2009 Jan;24(1):183-7. doi: 10.1007/s00467-008-0949-0. Epub 2008 Aug 8.
Advances in hematopoietic stem cell transplantation (HSCT) for beta-thalassemia major make the long-term outcome of these patients very important. Few data on long-term renal function of thalassemia patients are available. We evaluated the renal function in children after successful allogeneic HSCT for beta-thalassemia. Twenty-nine patients were included; the mean age at HSCT was 4.9 years. Mean follow-up time was 7.6 years. After HSCT, two patients developed acute renal failure and two had graft versus host disease. At last follow up, height standard deviation score (SDS) remained the same, but weight SDS had improved. Mean hemoglobin was 12.5 g/dl, and serum ferritin level was 545 ng/ml. All children had normal estimated glomerular filtration rate (GFR). One patient had hypertension and proteinuria, 10 years after HSCT. When comparing 39 children of the same age with beta-thalassemia of similar disease severity but who had not experienced HSCT, we found that the parameters of renal tubule function were better in patients that had undergone HSCT, as demonstrated by urine protein level (0.36 mg/mg creatinine vs 3.03 mg/mg creatinine, P < 0.001), osmolality (712 mosmol/kg vs 573 mosmol/kg, P = 0.006), N-acetyl-beta-D: -glucosaminidase (17.7 U/g creatinine vs 42.9 U/g creatinine, P = 0.045), and beta 2 microglobulin (0.09 microg/mg creatinine vs 0.13 microg/mg creatinine, P = 0.029). This study showed a low incidence of long-term renal impairment after HSCT and indicated that renal tubule function may be better in beta-thalassemia patients after HSCT.
重型β地中海贫血患者进行造血干细胞移植(HSCT)技术的进步使得这些患者的长期预后变得非常重要。目前关于地中海贫血患者长期肾功能的数据较少。我们评估了成功进行异基因HSCT治疗β地中海贫血的儿童的肾功能。共纳入29例患者;HSCT时的平均年龄为4.9岁。平均随访时间为7.6年。HSCT后,2例患者发生急性肾衰竭,2例发生移植物抗宿主病。在最后一次随访时,身高标准差评分(SDS)保持不变,但体重SDS有所改善。平均血红蛋白为12.5 g/dl,血清铁蛋白水平为545 ng/ml。所有儿童的估计肾小球滤过率(GFR)均正常。1例患者在HSCT后10年出现高血压和蛋白尿。当将39例年龄相同、疾病严重程度相似但未接受HSCT的β地中海贫血儿童进行比较时,我们发现接受HSCT的患者肾小管功能参数更好,尿蛋白水平(0.36 mg/mg肌酐vs 3.03 mg/mg肌酐,P < 0.001)、渗透压(712 mosmol/kg vs 573 mosmol/kg,P = 0.006)、N-乙酰-β-D-氨基葡萄糖苷酶(17.7 U/g肌酐vs 42.9 U/g肌酐,P = 0.045)和β2微球蛋白(0.09 μg/mg肌酐vs 0.13 μg/mg肌酐,P = 0.029)即可证明。本研究显示HSCT后长期肾功能损害的发生率较低,并表明HSCT后β地中海贫血患者的肾小管功能可能更好。